Data from First Clinical Trial of GGF2 in Heart Failure Presented at the American College of Cardiology 62nd Annual Scientific Session

ARDSLEY, N.Y. & NASHVILLE, Tenn.--(BUSINESS WIRE)--Mar 7,
2013 - Acorda Therapeutics, Inc. (Nasdaq:
ACOR) and collaborator Vanderbilt University Medical Center
today announced data from a Phase 1 clinical trial of Glial Growth
Factor 2 (GGF2) designed to study safety, tolerability and
exploratory measures of efficacy in people with heart failure who
were already on optimized regimens of currently available
therapies. The study evaluated the effects of a range of doses,
with each participant receiving a single dose. Data from this
trial, which enrolled patients at Vanderbilt and St. Joseph's
Hospital in Atlanta, GA, are being presented on Sunday, March 10 at
the American College of Cardiology 62nd Annual
Scientific Session in San Francisco, CA.

“We have completed the first in human trial with GGF2 in
patients with heart failure, and especially want to thank our
patients who volunteered for this important study. We are very
encouraged by the results,” said Daniel Lenihan, M.D.,
Professor of Medicine and Director, Clinical Research at the
Vanderbilt University Medical Center, Division of Cardiovascular
Medicine. “It is notable that trends of long-lasting and
dose-related improvement in cardiac function were seen following a
single dose in patients who were already optimized on standard
therapies. GGF2 warrants further investigation as a treatment for
heart failure.”

“Preclinical studies have suggested that GGF2 may improve
heart function through direct repair of cardiac muscle, a novel
mechanism of action. This first clinical trial in patients with
heart failure identified a maximally tolerated GGF2 dose and key
safety parameters to be monitored in future studies. This
information supports continued development of the compound as a
potential treatment for heart failure,” said Anthony
Caggiano, M.D., Ph.D., Vice President of Research and Development
at Acorda.

This was a double-blind, placebo controlled, escalating single
dose clinical trial that included 40 patients with advanced heart
failure. Safety and exploratory efficacy were monitored for 90 days
in patients randomized to receive various doses of GGF2 or
placebo.

Safety Findings

In this study, a single dose of GGF2 in patients with heart
failure was generally well tolerated up to 0.75 mg/kg.
Among participants receiving GGF2, the most commonly observed
adverse events were headache, site injection reaction and
gastrointestinal symptoms. There were no notable effects of
treatment on hematology or electrocardiogram, and no adverse events
led to withdrawal from the study.

A dose-limiting adverse event of hepatotoxicity (liver injury)
meeting Hy's Law criteria (elevated ALT, AST and bilirubin)
occurred in the highest-dose cohort. The patient's liver function
tests and bilirubin had returned to normal by two weeks after
dosing. There was also one reported case of uroepithelial
carcinoma, a form of cancer in the cells that line the bladder,
which was diagnosed three months after dosing in the highest-dose
cohort. The patient's baseline urinalysis showed the presence of
red blood cells, indicating that the tumor was likely present prior
to dosing.

Ejection Fraction Findings

A left ventricle ejection fraction of 55% or higher is
considered normal; all participants in the Phase 1 GGF2 trial had
left ventricle ejection fraction of less than 40%. Trial
participants receiving GGF2 showed a consistent and dose-responsive
trend towards improving left ventricular ejection fraction over 28
and 90 days compared to placebo.

Mean ejection fractions at screening in the treatment and
placebo groups were 27% and 29%, respectively. For the cohort
receiving the maximally tolerated dose (0.75 mg/kg) of GGF2, the
mean ejection fraction at screening was 28% and the absolute mean
changes in ejection fraction at day 8, day 14, day 28, and day 90
were 5%, 12%, 12.0% and 9.0%, compared to absolute mean changes of
-1%, -1%, 0% and 2% for the placebo group; thus, the mean ejection
fraction for this GGF2 group at day 28 was 40%, versus 29% for
placebo.

Acorda has discussed the findings from this initial study with
the U.S. Food and Drug Administration (FDA) and has reached
agreement on the next clinical study of GGF2 in heart failure. This
study will primarily investigate further the safety profile of GGF2
across a range of doses, and will continue to explore efficacy
outcomes.

The FDA has granted Fast Track designation for GGF2 for the
treatment of heart failure.

About GGF2

GGF2 is the leading development candidate from Acorda's
neuregulin program. Neuregulins are a class of naturally occurring
protein growth factors that have multiple effects on the nervous
and cardiovascular systems.

Preclinical studies demonstrate that GGF2 acts directly to
repair cardiac muscle and improve its contractile function. No
currently available therapies do this, and GGF2 may therefore offer
an important new treatment option for people with heart
failure.

In addition to its clinical program in heart failure, the
Company also has preclinical development programs for GGF2 in a
number of neurological indications, including peripheral nerve
injury and stroke.

Acorda Therapeutics is a biotechnology company focused on
developing therapies that restore function and improve the lives of
people with MS, spinal cord injury and other neurological
conditions.

Acorda markets
AMPYRA® (dalfampridine) Extended Release
Tablets, 10 mg, in the United States as a treatment to improve
walking in patients with multiple sclerosis (MS). This was
demonstrated by an improvement in walking speed. AMPYRA is marketed
outside the United States as FAMPYRA®
(prolonged-release fampridine tablets) by Biogen Idec under a
licensing agreement from Acorda. AMPYRA and FAMPYRA are
manufactured under license from Alkermes Pharma Ireland
Limited.

The Company also markets
ZANAFLEX CAPSULES® (tizanidine hydrochloride)
and Zanaflex tablets, a short-acting drug for the management of
spasticity. Acorda also receives sales royalties on tizanidine
hydrochloride capsules, an authorized generic version of ZANAFLEX
CAPSULES, distributed by Actavis, Inc. under its agreement with
Acorda.

Acorda has an industry-leading pipeline of novel neurological
therapies. The Company is developing Diazepam Nasal Spray for
treatment of certain epileptic seizures. It is also studying AMPYRA
to improve a range of functional impairments caused by MS, as well
as its potential for use in other neurological conditions,
including cerebral palsy and post-stroke deficits. In addition,
Acorda is developing clinical stage compounds AC105 for acute
treatment of spinal cord injury, GGF2 for treatment of heart
failure and rHIgM22, a remyelinating monoclonal antibody, for the
treatment of MS. GGF2 is also being investigated in
preclinical studies as a treatment for neurological conditions such
as stroke and spinal cord injury. Chondroitinase, an enzyme that
encourages nerve plasticity in spinal cord injury, is in
preclinical development.

Acorda Forward-Looking Statements

This press release includes forward-looking statements within
the meaning of the Private Securities Litigation Reform Act of
1995. All statements, other than statements of historical facts,
regarding management's expectations, beliefs, goals, plans or
prospects should be considered forward-looking. These statements
are subject to risks and uncertainties that could cause actual
results to differ materially, including our ability to successfully
market and sell Ampyra in the U.S.; third party payers (including
governmental agencies) may not reimburse for the use of Ampyra or
our other products at acceptable rates or at all and may impose
restrictive prior authorization requirements that limit or block
prescriptions; the risk of unfavorable results from future studies
of Ampyra or from our other research and development programs,
including Diazepam Nasal Spray or any other acquired or in-licensed
programs; we may not be able to complete development of, obtain
regulatory approval for, or successfully market Diazepam Nasal
Spray or other products under development; the occurrence of
adverse safety events with our products; delays in obtaining or
failure to obtain regulatory approval of or to successfully market
Fampyra outside of the U.S. and our dependence on our collaboration
partner Biogen Idec in connection therewith; competition,
including the impact of generic competition on Zanaflex Capsules
revenues; failure to protect our intellectual property, to defend
against the intellectual property claims of others or to obtain
third party intellectual property licenses needed for the
commercialization of our products; failure to comply with
regulatory requirements could result in adverse action by
regulatory agencies; and the ability to obtain additional financing
to support our operations. These and other risks are described in
greater detail in Acorda Therapeutics' filings with the
Securities & Exchange Commission. Acorda may not
actually achieve the goals or plans described in its
forward-looking statements, and investors should not place undue
reliance on these statements. Forward-looking statements made in
this release are made only as of the date hereof, and Acorda
disclaims any intent or obligation to update any forward-looking
statements as a result of developments occurring after the date of
this release.